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    Summary
    EudraCT Number:2021-004167-27
    Sponsor's Protocol Code Number:RM-493-033
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-03-02
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2021-004167-27
    A.3Full title of the trial
    A Phase 3 Multi-Center, One-Year, Open-Label study of Setmelanotide in Pediatric Patients Aged 2 to <6 years of age with Rare Genetic Causes of Obesity
    Estudio abierto de fase III, multicéntrico y de un año de setmelanotida en pacientes pediátricos de 2 a <6 años de edad con causas genéticas raras de obesidad
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to investigate treatment of Setmelanotide in children from 2 to 6 years old that have a form of genetic obesity
    Un estudio para investigar el tratamiento de Setmelanotida en niños de 2 a 6 años que tienen una forma de obesidad genética
    A.4.1Sponsor's protocol code numberRM-493-033
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation PlanP/215/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorRhythm Pharmaceuticals Limited
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportRhythm Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRhythm Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Associate
    B.5.3 Address:
    B.5.3.1Street Address222 Berkeley Street, 12th floor
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02116
    B.5.3.4CountryUnited States
    B.5.6E-mailccokkinias@rhythmtx.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Imcivree
    D.2.1.1.2Name of the Marketing Authorisation holderRhythm Pharmaceuticals Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/16/1703, EU/3/18/2101 and EU/3/19/2192
    D.3 Description of the IMP
    D.3.1Product namesetmelanotide
    D.3.2Product code RM-493
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSetmelanotide
    D.3.9.1CAS number 920014-72-8
    D.3.9.2Current sponsor codeRM-493
    D.3.9.3Other descriptive nameRM-493
    D.3.9.4EV Substance CodeSUB192416
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    POMC deficiency obesity due to mutations in the POMC gene
    PCSK1 deficiency obesity due to mutations in the PCSK1 gene
    LEPR deficiency obesity due to mutations in the LEPR gene
    Bardet-Biedl syndrome
    Obesidad por deficiencia de POMC debido a mutaciones en el gen POMC
    Deficiencia por obesidad de PCSK1 debido a mutaciones en el gen PCSK1
    Obesidad por deficiencia de LEPR debido a mutaciones en el gen LEPR
    Síndrome de Bardet-Biedl
    E.1.1.1Medical condition in easily understood language
    Obesity caused by rare genetic defects in patients causing extreme hunger.
    Obesidad causada por raros defectos genéticos en pacientes que provocan hambre extrema.
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.0
    E.1.2Level PT
    E.1.2Classification code 10084105
    E.1.2Term Leptin receptor deficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.0
    E.1.2Level PT
    E.1.2Classification code 10083937
    E.1.2Term Pro-opiomelanocortin deficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10048680
    E.1.2Term Bardet-Biedl syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of setmelanotide on weight in pediatric patients aged 2 to <6 years with obesity due to either (1) biallelic variants of the POMC, PCSK1 or LEPR genes or (2) Bardet-Biedl Syndrome (BBS) by determining if they meet a “responder” definition for change in body weight.
    Evaluar el efecto de la setmelanotida en el peso de pacientes pediátricos de 2 a <6 años con obesidad debido a (1) variantes bialélicas de los genes POMC, PCSK1 o LEPR o (2) síndrome de Bardet-Biedl (SBB) determinando si cumplen una definición de "paciente con respuesta" para el cambio en el peso corporal
    E.2.2Secondary objectives of the trial
    Secondary:
    To evaluate the effect of setmelanotide on weight in pediatric patients aged 2 to <6 years with obesity due to either (1) biallelic variants of the POMC, PCSK1 or LEPR genes or (2) BBS using additional measures of changes in body weight.

    To evaluate the safety and tolerability of setmelanotide in pediatric patients aged 2 to <6 years with obesity due to either (1) biallelic variants of the POMC, PCSK1 or LEPR genes or (2) BBS.

    Exploratory:
    To evaluate the effect of setmelanotide on (1) metabolic parameters, (2) waist circumference, (3) pharmacokinetics and (4) quality of life and care taker burden in pediatric patients between the ages of 2 to <6 years old with obesity due to either (1) biallelic variants of the POMC, PCSK1 or LEPR genes or (2) BBS.
    Objetivos Secundarios:
    • Evaluar el efecto de la setmelanotida en el peso de pacientes pediátricos de 2 a <6 años con obesidad debido a (1) variantes bialélicas de los genes POMC, PCSK1 o LEPR o (2) SBB mediante mediciones adicionales de cambios en el peso corporal.
    • Evaluar la seguridad y la tolerabilidad de la setmelanotida en pacientes pediátricos de 2 a <6 años con obesidad debido a (1) variantes bialélicas de los genes POMC, PCSK1 o LEPR o (2) SBB.

    Objetivo Exploratorio:
    • Evaluar el efecto de la setmelanotida sobre (1) los parámetros metabólicos, (2) el perímetro de la cintura, (3) la farmacocinética y (4) la calidad de vida y la carga del cuidador en pacientes pediátricos de 2 a <6 años con obesidad debido a (1) variantes bialélicas de los genes POMC, PCSK1 o LEPR o (2) SBB.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients must have obesity due to either:
    a. POMC, PCSK1, or LEPR deficiency, confirmed by genetic testing demonstrating biallelic variants that are interpreted as pathogenic, likely pathogenic, or of undetermined significance (VUS) by the American College of Medical Genetics and Genomics (ACMG) criteria, or
    b. BBS as defined by both (1) the Beales Criteria, 1999 (Beales 1999 [Appendix 16.1]) AND (2) genetic confirmation of homozygous or compound heterozygous loss-of-function mutation in BBS genes.

    2. Age between 2 to <6 years at the time of informed consent are eligible for the study.

    3. Obese, defined as body mass index (BMI) ≥97th percentile for age and gender AND body weight of at least 20 kg at the time of enrollment

    4. Parent or guardian of study participant is able to communicate well with the PI, to understand and comply with the requirements of the study (including once daily (QD) injection regimen and all other study procedures) and is able to understand and sign the written consent.
    1. Los pacientes deben tener obesidad debido a:
    a. Deficiencia de POMC, PCSK1 o LEPR, confirmada por pruebas genéticas que demuestran variantes bialélicas que se interpretan como patógenas, probablemente patógenas o de significación indeterminada (VSI) según los criterios del Colegio Americano de Genética Médica y Genómica (American College of Medical Genetics and Genomics, ACMG), o
    b. SBB según lo definido por (1) los Criterios de Beales, 1999 (Beales 1999 [Apéndice 16.1]) Y (2) la confirmación genética de la mutación de pérdida de función homocigótica o heterocigótica compuesta en genes de SBB.
    2. Los niños con edades entre 2 y <6 años en el momento del consentimiento informado son elegibles para el estudio.
    3. Obesidad, definida como índice de masa corporal (IMC)≥ percentil 97 para la edad y el sexo Y peso corporal de al menos 20 kg en el momento de la inscripción
    4. El progenitor o tutor del participante del estudio puede comunicarse bien con el IP, comprender y cumplir con los requisitos del estudio (incluida la pauta de inyección una vez al día [1 v/d] y todos los demás procedimientos del estudio) y puede comprender y firmar el consentimiento por escrito.
    E.4Principal exclusion criteria
    1. HbA1c >9.0% at screening

    2. History of significant liver disease other than non-alcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH).

    3. Glomerular filtration rate (GFR) <60 mL/min

    4. History or close family history (parents or siblings) of melanoma, or patient history of oculocutaneous albinism.

    5. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions (excluding non-invasive basal or squamous cell lesion), determined as part of a comprehensive skin evaluation performed by the PI during screening. Any concerning lesions identified during screening will be biopsied and results known to be benign prior to enrollment.

    6. Patient is, in the opinion of the Study PI, not suitable to participate in the study.

    7. Participation in any clinical study with an investigational drug/device within 3 months prior to the first day of dosing.

    8. Previously enrolled in a clinical study involving setmelanotide or any previous exposure to setmelanotide.

    9. Significant hypersensitivity to any excipient in the study drug.

    10. Inadequate hepatic function as evidenced by elevated Aspartate Aminotransferase (AST) and Alanine Aminotransferase (ALT) values >5x Upper limit of normal (ULN).
    1. HbA1c >9,0 % en la selección
    2. Antecedentes de enfermedad hepática significativa distinta de la enfermedad del hígado graso no alcohólico (EHGNA) o la esteatohepatitis no alcohólica (EHNA).
    3. Tasa de filtración glomerular (TFG) <60 ml/min
    4. Antecedentes o antecedentes familiares cercanos (padres o hermanos) de melanoma, o antecedentes personales de albinismo oculocutáneo.
    5. Hallazgos dermatológicos significativos relacionados con melanoma o lesiones cutáneas pre-melanoma (excluidas las lesiones de células epidermoides o basocelulares no invasivas), determinados como parte de una evaluación cutánea exhaustiva realizada por el IP durante la selección. Antes de la inscripción, se realizará una biopsia de cualquier lesión relacionada que se identifique durante la selección y los resultados deberán ser benignos.
    6. En opinión del IP del estudio, el paciente no es apto para participar en el estudio.
    7. Participación en cualquier estudio clínico con un medicamento/dispositivo en investigación dentro de los 3 meses anteriores al primer día de administración.
    8. Previamente inscrito en un estudio clínico con setmelanotida o cualquier exposición previa a setmelanotida.
    9. Hipersensibilidad significativa a cualquier excipiente del fármaco del estudio.
    10. Función hepática inadecuada evidenciada por valores elevados de aspartato aminotransferasa (AST) y alanina aminotransferasa (ALT) >5 veces el límite superior de la normalidad (LSN).
    E.5 End points
    E.5.1Primary end point(s)
    The response rate to setmelanotide, with a “responder” defined as a decrease from baseline in the patient’s BMI z-score of ≥0.2.
    La tasa de respuesta a la setmelanotida, con un "paciente con respuesta" definido como aquel con reducción desde el valor inicial en la puntuación z del IMC del paciente ≥0,2.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Patient age, height and weight will be recorded at each patient visit starting screening, enrollment, week 2, 4, 6, 8, 12, 16, 20, 28, 36, 44 and 52.
    La edad del paciente, la altura y el peso será registrada en cada visita del paciente incluyendo las visitas de cribado, inscripcion, semanas: 2, 4, 6, 8, 12, 16, 20, 28, 36, 44 y 52
    E.5.2Secondary end point(s)
    Secondary:
    The change in percent of the 95th percentile of body mass index (BMI), the actual and percent change in weight and the change in BMI and change in BMI-z score from baseline to the end of study will be summarized.

    Frequency and severity of AEs, vital signs, and change from baseline in bone age, Ages & Stages Questionnaires, Third Edition (ASQ®-3) and laboratory evaluations.

    Exploratory:
    The change from baseline in metabolic parameters (as measured by the fasting lipid profile and change in hemoglobin A1c), change in waist circumference from baseline, pharmacokinetic parameters, and change in caregiver burden (as measured by the Zarit Burden Interview instrument) and quality of life (as measured by the Work Productivity and Activity Impairment, PROMIS Global Health - Parent Proxy and Caregivers instruments) will be summarized.
    Criterios de valoración secundarios:
    • Se resumirán el cambio en el porcentaje del percentil 95 del índice de masa corporal (IMC), el cambio real y porcentual en el peso y el cambio en el IMC y el cambio en la puntuación z del IMC desde el inicio hasta el final del estudio.
    • Frecuencia e intensidad de AA, constantes vitales y cambio desde el inicio en la edad ósea, Cuestionarios de edades y estadios, Tercera edición (ASQ®-3) y evaluaciones de laboratorio.

    Criterios de valoración exploratorios:
    El cambio desde el inicio en los parámetros metabólicos (medidos por el perfil de lípidos en ayunas y el cambio en la hemoglobina A1c), el cambio en el perímetro de la cintura desde el inicio, los parámetros farmacocinéticos y el cambio en la carga del cuidador (medido por el instrumento de entrevista de carga de Zarit) y la calidad de vida (medida por los instrumentos de deterioro de la productividad laboral y la actividad y salud global de PROMIS para representantes de progenitores y cuidadores).
    E.5.2.1Timepoint(s) of evaluation of this end point
    End points are assessed from baseline to end of treatment.
    Los criterios de evaluación serán valorados des la línea basal y hasta el fin del tratamiento
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    United States
    Netherlands
    Spain
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last visit last subject
    Última visita del último paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months10
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 10
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children aged between 2 and <6 years of age.
    Niños de entre 2 y menores de 6 años
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 10
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    At the end of the study patients can choose to end the study or move directly to an active long-term extension study.
    Al final del estudio, los pacientes pueden elegir terminar el estudio o pasar directamente a un estudio de extensión activo a largo plazo.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-05-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-26
    P. End of Trial
    P.End of Trial StatusOngoing
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